The call for an EU action plan for rare diseases – POLITICO

From a funding perspective, we understand that governments face difficult ethical decisions about how best to allocate public resources to balance the healthcare needs of many versus the few. For ultra-rare diseases, a political decision to secure funding is imperative so that people with very rare diseases are not left out.

As an industry and as a continent, we in Europe continue to push scientific boundaries. We are adopting new clinical trial designs that target specific patient groups and allow for greater flexibility, more efficient data collection and faster decision-making. These approaches increase the chances of success in rare disease trials. The Rare Disease Moonshot initiative, supported by EFPIA, which uses public-private partnerships to accelerate research^(8th)is a fantastic example of this collaborative mindset in practice.

However, our approach remains inconsistent. The level of budget allocated to pharmaceutical spending varies considerably across Europe. In the Nordic countries, Belgium, the Netherlands, Ireland and Austria, the amount is between 6 and 12 percent of total health expenditure. In other countries, such as Greece, Poland, Hungary and Bulgaria, pharmaceutical spending is between 20 and 30 percent of total health expenditure. ⁽⁹⁾. These figures speak for themselves – they create the potential for significant disparities in reimbursement for new innovations and risk widening health inequalities for European citizens. Some Member States have early access programmes for new and innovative medicines, others do not. Some of these programmes are free, others are not. If the pharmaceutical industry continues to cover a large proportion of the costs of some of these early access programmes, how can we create incentives for payers to address the problem in a sustainable way? Europe must adopt an approach based on ability to pay, not willingness to pay.

EU competitiveness in life sciences: a double opportunity for the rare disease sector

An EU Action Plan for Rare Diseases is more than a political imperative; it is a moral obligation towards the millions of people living with a rare disease.

By joining forces with initiatives already underway such as the Rare Disease Moonshot, the European Joint Programme for Rare Diseases (ERDERA), Together4 Rare Diseases (T4RD) and Screen4Care, we can bridge the gap between potential and reality through a dynamic innovation ecosystem supported by an enabling policy framework and targeted investments. Together, we can turn the tide for rare disease patients and turn challenges into opportunities for breakthroughs that go beyond scientific laboratories and impact the lives of people with rare diseases – in the EU and beyond.